Chicago, Dec. 16, 2021 – Paragon Biosciences has had a stellar year of achievements in 2021, advancing our mission to solve some of society’s most challenging problems. From receiving positive topline study results and adding new potential therapies to pipelines, to launching new adaptive biology programs, Paragon and our seven portfolio companies, with several more in incubation, continue to innovate by accelerating biology-based scientific breakthroughs.

Paragon has shown great growth with nearly $500 million raised since January. Paragon and our partners have invested more than $1.3 billion dollars to support our portfolio companies since 2017, creating enormous value for patients and shareholders.

“Our work is important and meaningful, so it’s necessary to invest and create a culture of innovation,” said Jeff Aronin, founder, chairman and CEO, Paragon Biosciences. “Our accomplishments are a reflection of the exemplary teams we have assembled and their commitment to our mission.”

Following are several highlights of our impressive achievements in 2021:

Harmony Biosciences
Harmony Biosciences, (“Harmony”) (Nasdaq: HRMY), one of the earliest companies established by Paragon Biosciences, has had a series of achievements that position the company for continued growth in revenue and scientific innovation. To support the growth of its business, Harmony entered into a strategic financing collaboration with Blackstone (NYSE: BX) in August to provide the company with up to $330 million of financing and growth capital. Paragon Biosciences’ wholly owned subsidiary, Paragon Health Capital, conducted a highly competitive process to assist Harmony in achieving its objectives.

The company’s lead product, WAKIX® (pitolisant), is approved in the U.S. for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. The company released its strong first quarter, second quarter and third quarter financial results and business updates, reporting positive net income and profitability for all three quarters. Net product revenues increased for each quarter, when compared to the same period in 2020.

Related to its therapeutic pipeline, on Dec. 1, the company announced that the U.S. Food and Drug Administration (FDA) has accepted an Investigational New Drug (IND) application for pitolisant for the treatment of idiopathic hypersomnia (IH). Harmony is planning to initiate a Phase 3 clinical trial to evaluate the safety and efficacy of pitolisant in adult patients with IH in the first half of 2022.

Harmony also has initiated a Phase 2 clinical trial evaluating the safety and efficacy of a therapy for EDS and other non-muscular symptoms in adult patients with type 1 myotonic dystrophy (DM1), the most common form of adult-onset muscular dystrophy.

Harmony is also conducting a Phase 2 clinical trial evaluating the safety and efficacy of pitolisant for the treatment of EDS and other key symptoms in patients with Prader-Willi syndrome (PWS).

In addition, Harmony acquired a potential first-in-class molecule with a novel mechanism of action from ConSynance Therapeutics, Inc., a clinical stage biotechnology company focused on rare central nervous system diseases. The asset builds on Harmony’s commitment to developing safe and effective therapies for people living with rare diseases with high unmet medical needs.

Emalex Biosciences
Most recently, Emalex Biosciences, founded by Paragon to develop treatments for central nervous system movement disorders and fluency disorders, announced positive topline results from its Phase 2b clinical study (D1AMOND Study). The study evaluated the efficacy and safety of ecopipam (EBS-101), an investigational, first-in-class, dopamine-1 (D1) receptor antagonist, for the treatment of pediatric patients with Tourette Syndrome. These positive results provide Emalex with momentum to continue its work on this potential treatment for pediatric Tourette’s patients.

A second study being conducted by Emalex using EBS-101 to treat childhood-onset fluency disorder, or stuttering, in adult patients is now fully enrolled. Results for this Phase 2 clinical trial are expected in the first half of 2022.

To support the critical work of Emalex with these clinical trials, the company completed a $35 million raise in March led by Paragon Biosciences, with Fidelity Management & Research Company LLC and Valor Equity Partners joining the round.

Castle Creek Biosciences
Looking to expand its cell and gene therapy platform for patients suffering from rare diseases with few or no treatment options, Castle Creek Biosciences announced in September a research collaboration with Mayo Clinic to advance discovery and pre-clinical development of investigational gene therapy candidates for the treatment of osteogenesis imperfecta and classical Ehlers-Danlos syndrome. Just one month later, the company was awarded a $1.825 million research grant over four years to support a meaningful portion of its Phase 3 clinical development of dabocemagene autoficel (FCX-007, D-Fi), an investigational gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).

Our revolutionary adaptive biology portfolio company, Evozyne, continues to make impressive strides at its 30,000-square-foot headquarters in Chicago’s Lincoln Park neighborhood. Evozyne combines the complexity of nature with machine learning to create optimized proteins with extraordinary impact. These proteins have the potential to solve some of society’s most intractable problems, like finding therapeutic solutions to disease processes and environmentally friendly energy sources.

Several strategic partnerships, including a collaboration with Takeda, and numerous projects and internal products are underway. The company has added top talent from notable universities and institutions nationwide in a variety of key areas and has grown to employ nearly 75 people in under a year. To support its important work, Evozyne closed a nearly $60 million Series A raise earlier this year, upsized 18% over the initial target of $50 million and oversubscribed multiple times.

CiRC Biosciences
CiRC Biosciences is currently in pre-clinical development of vision restoration therapies to treat retinitis pigmentosa (RP) and geographic atrophy (age-related macular degeneration, dry AMD).

This latest Paragon portfolio company was granted U.S. FDA Orphan Drug Designation in March for a cell therapy—chemically induced photoreceptor-like cells (CiPCs)—to treat RP, a genetic disorder affecting roughly 1 in 4,000 people. A preclinical study using a mouse model found that CiPCs were able to partially re-establish the pupillary reflex and demonstrate behavioral/functional improvement. The journal Nature published findings from this research study in 2020.

In June, Sai Chavala, M.D., CiRC’s co-founder and chief scientific officer, was awarded the 2021 Ludwig von Sallmann Clinician-Scientist Award from the Association for Research in Vision and Ophthalmology (ARVO) Foundation for Eye Research. Dr. Chavala was honored for his innovative accomplishments as a young clinician-scientist in the field of vision and ophthalmology.

Great Place to Work Certification
Capping off an exceptional year, Paragon recently announced that we have been certified as a Great Place to Work®. This prestigious distinction is entirely survey based on what current employees say about their experience working at Paragon.

Great Place to Work is the global authority on workplace culture, employee experience, and the leadership behaviors proven to deliver market-leading revenue, employee retention and increased innovation.

According to Great Place to Work research, job seekers are 4.5 times more likely to find a great boss at a Certified great workplace. Additionally, employees at Certified workplaces are 93% more likely to look forward to coming to work, and are twice as likely to be paid fairly, earn a fair share of the company’s profits and have a fair chance at promotion.


Product Safety Statements
About WAKIX® (pitolisant) Tablets

WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of EDS and cataplexy in adult patients with narcolepsy and has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010, fast track designations for the treatment of EDS and cataplexy in patients with narcolepsy in 2017, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H3) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H3 receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

Important Safety Information
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

Warnings and Precautions
WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information). WAKIX is not recommended in patients with end-stage renal disease (ESRD).

Adverse Reactions
In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash. To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or FDA at 1-800-FDA-1088 or

Please see the Full Prescribing Information for WAKIX for more information.

About Ecopipam
Ecopipam is an investigational first-in-class drug being evaluated for the treatment of Tourette Syndrome (TS) in pediatric patients and childhood-onset fluency disorder (stuttering) in adults. Ecopipam selectively blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1-receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with TS.

Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with TS. Adverse events affecting primarily the central nervous system [CNS] (e.g., sedation, insomnia, psychiatric changes) and the gastrointestinal system (e.g., nausea and vomiting) are the most frequently reported side effects from clinical studies.

About FCX-007
Castle Creek Biosciences’ dabocemagene autoficel (FCX-007, D-Fi) is being developed to address the deficiency of functional type VII collagen protein (COL7) in patients with dystrophic epidermolysis bullosa (DEB). FCX-007 is comprised of autologously-derived dermal fibroblasts genetically modified with a lentiviral vector containing the COL7A1 gene to express COL7. FCX-007 is locally administered by injection directly into the papillary dermis of persistent and non-healing recurrent wounds of DEB where the COL7 protein can support the formation of anchoring fibrils in the skin.

About the Phase 3 Study of FCX-007 (DeFi-RDEB)
DeFi-RDEB is a multi-center, within-patient randomized, controlled, open-label, Phase 3 clinical trial of FCX-007 designed to enroll approximately 24 people living with RDEB. Each participant’s target wounds are paired and then randomized to receive FCX-007 or remain untreated. Up to three target wound pairs are identified for each participant. The primary outcome measure is complete wound closure of the first wound pair at week 24. Currently, there are five trial locations recruiting participants including Stanford University in Stanford, California; Children’s Hospital Colorado in Aurora, Colorado; Dell Children’s Medical Group in Austin, Texas; Solutions Through Advanced Research, Inc. in Jacksonville, Florida; and Mayo Clinic in Rochester, Minnesota. More information about the Phase 3 trial is available at and by searching the identifier NCT04213261, and also at

Emalex Biosciences Expands CNS Platform to Advance Treatment for Restless Legs Syndrome with Augmentation